Amgen Sees Growth in Rare Disease Franchise

Rare Daily Staff

Amgen, which completed its $27.8 billion acquisition of Horizon Therapeutics in October, told analysts last week that it sees significant opportunities to grow its rare disease franchise beyond the nearly $4 billion business it represented in 2023.

On a call with analysts last week, Murdo Gordon, Amgen’s executive vice president of global commercial operations, said that the company sales to date are mostly in the United States. “Contributions from international expansion will be additive,’ he said. “We still see significant opportunity within the U.S. given that there is relatively low penetration for each of these products.”

Amgen’s move into rare disease began in late 2022 when the company completed its acquisition of ChemoCentryx, which produced Tavenos, a first-in-class medicine for patients with the rare, autoimmune disease ANCA-associated vasculitis.

“We’re leveraging our strong commercial and medical leadership in inflammation and nephrology along with our global scale to successfully launch and expand our rare disease products in key markets,” said Gordon, who said the company’s strategic focus on rare disease will “accelerate revenue growth” starting in 2024.

Two of the products Amgen gained through the acquisition of Horizon—Tepezza for thyroid eye disease and Uplizna for neuromyelitis optica spectrum disorder—offer particular opportunities for growth.

Vikram Karnani noted that phase 4 data on Tepezza demonstrated its benefits to a wider group of people with thyroid eye disease and in April 2023, the FDA expanded its approved use to treat thyroid eye disease of any duration or activity level.

“With this updated indication and the emphasis on low activity, longer duration TED patients, we see an opportunity to substantially grow the U.S. TED market,” said Vikram Karnani, executive vice president of  global commercial and medical affairs in rare disease for Amgen. There are about 100,000 patients with moderate to severe disease in the United States who could benefit from Tepezza, and Karnani said most of these patients have low clinical activity scores.

He noted that Tepezza penetration is in the high single digits in the TED population today. The new clinical data and the large portion of patients not currently treated with Tepezza gives Amgen confidence that there are significant growth opportunities in the United States for the drug.

He said Amgen’s focus this year will be to accelerate education efforts to reach new prescribers and target ophthalmologists and endocrinologists, who tend to treat patients with lower clinical activity.

With regard to Uplizna, he said Amgen has a significant opportunity to reach thousands of patients by looking beyond the United States. In Europe, where the company won approval in April 2022, there are an estimated 8,000 to 10,000 addressable patients. The company sees similar potential in Brazil, where the company won approval in December 2022. And finally, in Canada, Uplizna won approval in January this year.

“This global expansion is a critical part of Uplizna’s long-term growth strategy and a key area that we are already realizing the benefits of the acquisition to deliver results,” Karnani said.

Photo: Vikram Karnani, executive vice president of  global commercial and medical affairs in rare disease for Amgen